CTIS2023-503770-21-00进行中（未招募）1 期
A Randomized, Double-Blind, Placebo-Controlled, Dose-Ranging Study to Evaluate the Efficacy and Safety of SPR001 (Tildacerfont) in Adult Subjects with Classic Congenital Adrenal Hyperplasia - SPR001-203
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 75
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 年龄范围
- 18 至 65+（—）
- 性别
- All
入选标准
- •1.Male and female subjects = 18 years old at screening., 2.Has a known childhood diagnosis of classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency based on genetic mutation in CYP21A2 and/or documented (at any time) elevated 17-hydroxyprogesterone and currently treated with hydrocortisone, hydrocortisone acetate, prednisone, prednisolone, methylprednisolone (or a combination of the aforementioned glucocorticoids)., 3. Has been on a stable, supraphysiologic dose of glucocorticoid replacement for =1 month before screening., 4. Has androstenedione >upper limit of normal at both screening and Week 4 if daily glucocorticoid dose <30 mg OR has androstenedione >2.5x upper limit of normal at both screening and Week 4., 5.For subjects with the salt-wasting form of congenital adrenal hyperplasia, the subject is on a stable dose of mineralocorticoid replacement for =1 month before screening., 6.Agrees to follow contraception guidelines. Male subjects must also agree to refrain from donating sperm throughout the treatment period and for 90 days after the last dose of study drug., 7. Is able to understand all study procedures and risks involved and provides written informed consent indicating willingness to comply with all aspects of the protocol.
排除标准
- •1.Has a known or suspected diagnosis of any other known form of classic congenital adrenal hyperplasia (not due to 21-hydroxylase deficiency)., 7.c.Current or history of liver disease (with the exception of Gilbert’s syndrome)., 7.d.History of alcohol or substance abuse within the last year, or any significant history of alcohol or substance abuse that would likely prevent the subject from reliably participating in the study, based on the opinion of the Investigator., 7.e.Active hepatitis B, hepatitis C, or human immunodeficiency virus at screening., 7.f. Subjects who plan to undergo bariatric surgery during the study are excluded., 7.g.Any other condition that would impact subject safety or confound interpretation of study results., 8.Psychiatric conditions, including but not limited to depression, bipolar disorder, schizophrenia or schizoaffective disorder that are not effectively controlled on medication and may have an adverse impact on study compliance. Symptoms including hallucinations, delusions and psychosis are exlcusionary., 8.a.Increased risk of suicide based on the Investigator's judgment or the results of the Columbia–Suicide Severity Rating Scale (C-SSRS) conducted at screening and Week 6 (eg, C-SSRS Type 3, 4, or 5 ideation within the past 6 months or any suicidal behavior within the past 12 months), 8.b.Hospital Anxiety and Depression Scale score >12 for either depression or anxiety at screening or Week 6., 9. Has clinically significant abnormal ECG or clinical laboratory results., 10. Routinely works overnight shifts., 2.Has a history that includes bilateral adrenalectomy or hypopituitarism., 11. Subjects with travel plans/work schedules that result in significant and frequent changes in time zones (>2 hours) will require Medical Monitor approval for enrollment., 12.Females who are pregnant or nursing., 13.Use of any other investigational drug from 30 days or 5 half-lives (whichever is longer) before screening to the end of the study., 14.Use of the following drugs from 30 days or 5 half-lives (whichever is longer) before Day 1 to the end of the study:, 14.a.rosiglitazone, aromatase inhibitors, testosterone, or growth hormones or any other medication or supplement that could impact subject safety or confound interpretation of study results., 14.b.drugs listed in the study protocol., 15.Donation or receipt of blood from 90 days before Screening to the end of the study; donation or receipt of platelets, white blood cells, or plasma from 30 days before Screening to the end of the study., 3.Has a history of allergy or hypersensitivity to tildacerfont, or any of its excipients or any other CRF1 receptor antagonist., 4.Current treatment with dexamethasone as glucocorticoid therapy for congenital adrenal hyperplasia., 5.Is not adherent to glucocorticoid or study drug dosing regimen during the Run-in Period (defined as taking <80% of expected doses based on drug accountability)., 6.Shows clinical signs or symptoms of adrenal insufficiency., 7.Has had a clinically significant unstable medical condition, medically significant illness, or chronic disease occurring within 30 days of screening, including but not limited to:, 7.a. An ongoing malignancy or <3 years of remission history from any malignancy, other than successfully treated localized skin cancer., 7.b. Estimated glomerular filtration rate of <45 mL/min/1.73 m2.
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